NCT01548729已完成1 期
Metabolic Efficiency of Combined Pancreatic Islet and Lung Transplant for the Treatment of End-Stage Cystic Fibrosis : a Pilot Study
University Hospital, Strasbourg, France56 个研究点 分布在 1 个国家目标入组 14 人开始时间: 2012年2月25日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 14
- 试验地点
- 56
- 主要终点
- Metabolic efficiency at 1 year
研究概览
简要总结
Patients with end-stage cystic fibrosis (CF) and severe CF-related diabetes (CFRD) may benefit from combined lung-pancreatic islet transplantation. A recent case series showed that combined bilateral lung and pancreatic islet transplantation is a viable therapeutic option for patients with end-stage CF and CFRD. The use of different organs from a single donor may lead to reduced immunogenicity. As the prevalence of CFRD has increased dramatically with the improved life expectancy of patients with CF, islet transplantation should be considered at the end-stage CF. By restoring metabolic control, the investigators hypothesize that islet transplantation may improve the management of CF patients undergoing lung transplant and decrease the complication rate in the early postoperative period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 14 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient with cystic fibrosis
- •Patient able to respect the protocol procedures
- •Patient with end-stage respiratory insufficiency indicating a lung transplant
- •Clinical history of cystic fibrosis related diabetes and/or insulin-dependent diabetes, with no residual insulin secretion (C-peptide < 0,5 ng/mL) and/or no response to IV glucagon stimulation: [peak stimulated C-peptide (T6min)/basal plasma C-peptide(T0)] <
- •The absence of insulin secretion will be verified 2 times before inclusion
- •Evolution of diabetes for over 3 years
- •Patient whose glycaemic control obtained with insulin therapy is not satisfactory and could significantly alter quality of life (HbA1c > 7% and/or MAGE index > 1,25). This situation is assessed by a diabetologist.
- •Social Security membership or benefit from Social Welfare
- •Patient who received the results of the medical evaluation required
- •Non-inclusion criteria:
- •Patient with contra-indication for undergo a lung transplant
- •Patient with an indication of heart, liver or kidney transplantation
- •Patient for which poor therapeutic compliance is expected
- •Patient under oral antidiabetic drug
- •In women of childbearing potential: pregnancy test (urine and / or blood) positive, lactation, or unwilling to use effective contraception for the duration of the study until 3 months after the end . Men: procreation project during the study period up to 3 months after its end, or unwilling to use effective contraception patient.
- •Active infection, including hepatitis B, hepatitis C, HIV
- •Any history of malignancy within the past 5 years, with the exception of cutaneous basal cell carcinomas completely resected. The history of breast or melanoma cancers are an absolute contraindication
- •Alcoholic intoxication or drug addiction
- •Anemia (hemoglobin Hb <10g / dL in women and Hb <11 g / dL in men), lymphopenia (<1000 / uL), neutropenia (<1500 / uL) or thrombocytopenia (<100,000 / uL).
- •Persistent elevated liver enzymes at baseline
- •Portal hypertension identified by oesophageal varice and/or hypersplenism (platelets < 120 000 /mm3) or Child-Pugh score >
- •Use of a medical treatment under investigation within 4 weeks before inclusion
- •All medical situation assessed by an investigator which could interfere with the good management of the project
- •Patient restricted of freedom or unable to give his consent
- •Patient has been included in another study that could interfere with the results of the study
- •Contraindications to the use of experimental drugs (Cellcept®, prednisone, Prograf, prednisolone, methylprednisolone, and pancreatic islets)
排除标准
- 未提供
结局指标
主要结局
Metabolic efficiency at 1 year
时间窗: 1 year
Combined criteria based on the 4 following criteria: weight increase \> 5% compared to inclusion, fasting blood glucose \< 1.1 g/l at 12 months post-transplant (beta score criterion), Reducing insulin requirements (expressed in UI/day) compared to inclusion \& decreased in HbA1c \>= 0.5% (in absolute value) compared to inclusion Success is defined by achieving at least three of these criteria
次要结局
- Insulin requirements(Every month during 1 year after transplant)
- Ratio [C-peptide stimulated T6min/ C-peptide basal T0](Every 3 months during 1 year after transplant)
- Tiffeneau-Pinelli index(Every month during 1 year after transplant)
- Nature of pulmonary infection episodes and nature of acute lung rejection if need be(Every month during 1 year after transplant)
- Oxygen saturation SaO2 room air(Every month during 1 year after transplant)
- Adverse events(Every visit during 1 year after transplant)
- C-peptide stimulated by glucagon(Every 3 months during 1 year after transplant)
- Number of minor hypoglycemia(Every month during 1 year after transplant)
- Forced Vital Capacity (FVC)(Every month during 1 year after transplant)
- Microalbuminuria & proteinuria(Every 3 months during 1 year after transplant)
- Median maximum expiration flow(Every month during 1 year after transplant)
- Number of days of post-transplant hospitalization and during the follow-up(Every visit during 1 year after transplant)
- Forced Expiratory Volume (FEV1)(Every month during 1 year after transplant)
- Number of episodes of pulmonary rejection(Every visit during 1 year after transplant)
- Mortality(Every visit during 1 year after transplant)
- Ratio [C-peptide/Glucose-Creatinine] & ratio [C-peptide/Glucose](Every week during the first month, and every month during 1 year)
- HbA1c(Every 3 months during 1 year after transplant)
- Number of major hypoglycemia(Every month during 1 year after transplant)
- Glycemic variability (MAGE)(Every 6 months during 1 year after transplant)
- duration of hypoglycemia(Every 6 months during 1 year after transplant)
- Dyspnea score according to the mMRC scale(Every month during 1 year after transplant)
研究者
研究点 (56)
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